Track­ing a suc­cess­ful DL­B­CL as­sault in Phase II, Mor­phoSys sets out to seize quick OK for MOR208

En­cour­aged by a break­through des­ig­na­tion at the FDA and its lat­est batch of up­beat da­ta from a Phase II study of its an­ti-CD19 an­ti­body MOR208, Mor­phoSys says it’s blaz­ing a straight path to reg­u­la­tors in search of an ac­cel­er­at­ed ap­proval.

Mor­phoSys ex­ecs spent a good deal of time talk­ing to an­a­lysts yes­ter­day about their rea­sons for shoot­ing at a quick OK. The Ger­man biotech high­light­ed up­dat­ed da­ta demon­strat­ing that of the 68 evalu­able pa­tients in their study for dif­fuse large B cell lym­phoma — a pop­u­lar dis­ease tar­get in on­col­o­gy — they tracked a 49% over­all re­sponse rate and a 31% com­plete re­sponse rate.

They al­so have a pre­lim­i­nary pro­gres­sion free sur­vival rate of 50.5% at 12 months.

Malte Pe­ters

This is a sin­gle arm study of the Mor­phoSys an­ti­body com­bined with lenalido­mide for 81 DL­B­CL pa­tients not el­i­gi­ble for high-dose chemo or au­tol­o­gous stem cell trans­plan­ta­tion. And it says a lot about why biotechs are bar­rel­ing in­to can­cer with high ex­pec­ta­tions of break­ing new ground ear­li­er than ever.

Malte Pe­ters, the chief de­vel­op­ment of­fi­cer at Mor­phoSys, spelled it out for an­a­lysts:

Specif­i­cal­ly 29 out of 33 pa­tients which have re­spond­ed, that amount to 88% are on­go­ing, 20 of whom have an on­go­ing com­plete re­sponse. The mean time to re­spond was short with 1.8 months and the mean time to ex­pe­ri­ence a com­plete re­sponse was 3.6 months….

At this time, we are hop­ing to be able to sub­mit da­ta from the cur­rent L-MIND tri­al to the FDA as a ba­sis for reg­u­la­to­ry ap­proval. Our goal is to achieve an ac­cel­er­at­ed ap­proval based pri­mar­i­ly on the L-MIND study. Apart from L-MIND, we are cur­rent­ly in­ves­ti­gat­ing MOR208 in the same pa­tient pop­u­la­tion in our Phase III B-MIND tri­al. This is a head-to-head study in­ves­ti­gat­ing the ef­fi­ca­cy of MOR208 plus ben­damus­tine ver­sus rit­ux­imab plus ben­damus­tine. Rit­ux­imab plus ben­damus­tine is one of the most com­mon­ly used reg­i­mens in the re­lapse re­frac­to­ry DL­B­CL set­ting.

The FDA al­ready has this drug down in the spe­cial BTD path based on an ear­li­er snap­shot of da­ta from the same study. That gives Mor­phoSys a rea­son­able hope that it can move fast to­ward a fil­ing while lin­ing up the big­ger Phase III as a con­fir­ma­to­ry study.

Si­mon Mo­roney

At this point, the ac­cel­er­at­ed ap­proval path­way has been blazed by a mob of de­vel­op­ers who need­ed to show that they were do­ing a rea­son­able per­cent­age of late-stage pa­tients some good. The FDA has re­spond­ed with alacrity, and reg­u­la­tors in the US are on­ly pick­ing up even more speed un­der the di­rec­tion of FDA com­mis­sion­er Scott Got­tlieb. Mor­phoSys – al­lied with a group of A-list part­ners on oth­er drugs – has good rea­son to be­lieve that they can take the same short cut.

Not­ed CEO Si­mon Mo­roney:

“We are very en­cour­aged by our most re­cent clin­i­cal da­ta from the on­go­ing L-MIND tri­al, which sup­port our plan to de­vel­op MOR208 in com­bi­na­tion with lenalido­mide as a chemo-free treat­ment op­tion for this pa­tient pop­u­la­tion.”

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

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Stéphane Bancel at the Endpoints #JPM20 breakfast panel in San Francisco, January 2020 (Photo: Jeff Rumans, Endpoints News)

Mod­er­na says Covid-19 vac­cine boost­er in­creased im­mune re­sponse against vari­ants of con­cern

About a month after announcing their variant-specific Covid-19 vaccine boosters showed promising results in mice, Moderna says it now has some human data to back it up.

Volunteers given a booster shot about six to eight months after receiving their second dose saw increased antibody levels against SARS-CoV-2 and two variants of concern: B.1.351, which was first identified in South Africa, and P.1, first identified in Brazil, the company said on Wednesday.

FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

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Joe Wiley, Amryt CEO

A biotech with a yen for pricey rare dis­ease drugs — and bar­gain base­ment shop­ping — adopts an­oth­er or­phan in lat­est M&A pact

After making it through a long, painful haul to get past a CRL and on to an FDA approval last summer, little Chiasma has found a buyer.

Amryt $AMYT, a company known for its appetite for acquiring expensive drugs for rare diseases at bargain prices, snagged Chiasma and its acromegaly drug Mycapssa (octreotide) capsules in an all-stock deal — with an exchange of 0.396 shares of Amryt for every share of Chiasma.

Ngozi Okonjo-Iweala, Director general of WTO (AP Photo/Keystone/Alessandro Della Bella)

Opin­ion: Waiv­ing Covid-19 vac­cine IP could save lives, but where is the man­u­fac­tur­ing ca­pac­i­ty?

Droves of House Democrats in Washington and members of the European Parliament have now glommed onto a major push by India and South Africa at the WTO to abolish all IP around Covid-19 vaccines.

At first blush, waiving this IP sounds like an easy win: More Covid-19 vaccines made locally for more people means more lives saved. Simple enough, especially as low-income countries have received just a tiny fraction of the world’s vaccine allotment so far.